AU2016225178B2 - Materials and methods for treatment of hemoglobinopathies - Google Patents
Materials and methods for treatment of hemoglobinopathies Download PDFInfo
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- AU2016225178B2 AU2016225178B2 AU2016225178A AU2016225178A AU2016225178B2 AU 2016225178 B2 AU2016225178 B2 AU 2016225178B2 AU 2016225178 A AU2016225178 A AU 2016225178A AU 2016225178 A AU2016225178 A AU 2016225178A AU 2016225178 B2 AU2016225178 B2 AU 2016225178B2
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/48—Reproductive organs
- A61K35/54—Ovaries; Ova; Ovules; Embryos; Foetal cells; Germ cells
- A61K35/545—Embryonic stem cells; Pluripotent stem cells; Induced pluripotent stem cells; Uncharacterised stem cells
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/41—Porphyrin- or corrin-ring-containing peptides
- A61K38/42—Haemoglobins; Myoglobins
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/06—Antianaemics
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0634—Cells from the blood or the immune system
- C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0696—Artificially induced pluripotent stem cells, e.g. iPS
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2501/00—Active agents used in cell culture processes, e.g. differentation
- C12N2501/70—Enzymes
- C12N2501/73—Hydrolases (EC 3.)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2510/00—Genetically modified cells
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- Developmental Biology & Embryology (AREA)
- Cell Biology (AREA)
- General Engineering & Computer Science (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Immunology (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Hematology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Reproductive Health (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Gynecology & Obstetrics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Diabetes (AREA)
- General Chemical & Material Sciences (AREA)
- Crystallography & Structural Chemistry (AREA)
- Transplantation (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562119754P | 2015-02-23 | 2015-02-23 | |
| US62/119,754 | 2015-02-23 | ||
| PCT/IB2016/000276 WO2016135557A2 (fr) | 2015-02-23 | 2016-02-23 | Matériels et méthodes pour le traitement des hémoglobinopathies |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| AU2016225178A1 AU2016225178A1 (en) | 2017-08-31 |
| AU2016225178A9 AU2016225178A9 (en) | 2022-05-05 |
| AU2016225178B2 true AU2016225178B2 (en) | 2022-05-05 |
Family
ID=55795007
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2016225178A Ceased AU2016225178B2 (en) | 2015-02-23 | 2016-02-23 | Materials and methods for treatment of hemoglobinopathies |
| AU2016225179A Ceased AU2016225179C1 (en) | 2015-02-23 | 2016-02-23 | Materials and methods for treatment of hemoglobinopathies |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2016225179A Ceased AU2016225179C1 (en) | 2015-02-23 | 2016-02-23 | Materials and methods for treatment of hemoglobinopathies |
Country Status (8)
| Country | Link |
|---|---|
| US (4) | US20180030438A1 (fr) |
| EP (2) | EP3262172A2 (fr) |
| CN (2) | CN107532168A (fr) |
| AU (2) | AU2016225178B2 (fr) |
| BR (2) | BR112017017812A2 (fr) |
| CA (1) | CA2977455A1 (fr) |
| SG (2) | SG11201706766WA (fr) |
| WO (2) | WO2016135558A2 (fr) |
Families Citing this family (73)
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| US10323236B2 (en) | 2011-07-22 | 2019-06-18 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| DK3456831T3 (da) | 2013-04-16 | 2021-09-06 | Regeneron Pharma | Målrettet modifikation af rottegenom |
| US9163284B2 (en) | 2013-08-09 | 2015-10-20 | President And Fellows Of Harvard College | Methods for identifying a target site of a Cas9 nuclease |
| US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| KR102170502B1 (ko) | 2013-12-11 | 2020-10-28 | 리제너론 파마슈티칼스 인코포레이티드 | 게놈의 표적화된 변형을 위한 방법 및 조성물 |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| EP3122880B1 (fr) | 2014-03-26 | 2021-05-05 | Editas Medicine, Inc. | Méthodes liées à crispr/cas et compositions pour le traitement de la drépanocytose |
| US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
| KR102531016B1 (ko) | 2014-11-21 | 2023-05-10 | 리제너론 파마슈티칼스 인코포레이티드 | 쌍 형성된 가이드 rna를 사용하는 표적화된 유전자 변형을 위한 방법 및 조성물 |
| WO2016135559A2 (fr) | 2015-02-23 | 2016-09-01 | Crispr Therapeutics Ag | Matériaux et procédés pour le traitement de maladies génétiques humaines, y compris d'hémoglobinopathies |
| BR112017017812A2 (en) | 2015-02-23 | 2018-04-10 | Crispr Therapeutics Ag | Materials and methods for treatment of hemoglobinopathies |
| CA2986310A1 (fr) | 2015-05-11 | 2016-11-17 | Editas Medicine, Inc. | Systemes crispr/cas9 optimises et procedes d'edition de genes dans des cellules souches |
| AU2016261927B2 (en) * | 2015-05-12 | 2022-04-07 | Sangamo Therapeutics, Inc. | Nuclease-mediated regulation of gene expression |
| CN108026526B (zh) | 2015-06-09 | 2023-05-12 | 爱迪塔斯医药公司 | 用于改善移植的crispr/cas相关方法和组合物 |
| WO2017053729A1 (fr) | 2015-09-25 | 2017-03-30 | The Board Of Trustees Of The Leland Stanford Junior University | Édition du génome à médiation par une nucléase de cellules primaires et leur enrichissement |
| IL310721B2 (en) | 2015-10-23 | 2025-11-01 | Harvard College | Nucleobase editors and uses thereof |
| US12043843B2 (en) | 2015-11-04 | 2024-07-23 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of hemoglobinopathies |
| JP2019508051A (ja) * | 2016-03-14 | 2019-03-28 | エディタス・メディシン、インコーポレイテッド | β異常ヘモグロビン症を治療するためのCRISPR/CAS関連方法および組成物 |
| CN113831407B (zh) | 2016-05-20 | 2024-06-11 | 瑞泽恩制药公司 | 用于使用多个引导rna来破坏免疫耐受性的方法 |
| CN110214183A (zh) | 2016-08-03 | 2019-09-06 | 哈佛大学的校长及成员们 | 腺苷核碱基编辑器及其用途 |
| WO2018031683A1 (fr) | 2016-08-09 | 2018-02-15 | President And Fellows Of Harvard College | Protéines de fusion cas9-recombinase programmables et utilisations associées |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| KR102622411B1 (ko) | 2016-10-14 | 2024-01-10 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵염기 에디터의 aav 전달 |
| WO2018119359A1 (fr) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Édition du gène récepteur ccr5 pour protéger contre l'infection par le vih |
| WO2018140934A1 (fr) | 2017-01-30 | 2018-08-02 | The Children's Hospital Of Philadelphia | Compositions et méthodes de production d'hémoglobine |
| TW201839136A (zh) | 2017-02-06 | 2018-11-01 | 瑞士商諾華公司 | 治療血色素異常症之組合物及方法 |
| EP3592853A1 (fr) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression de la douleur par édition de gène |
| US12390514B2 (en) | 2017-03-09 | 2025-08-19 | President And Fellows Of Harvard College | Cancer vaccine |
| US11542496B2 (en) | 2017-03-10 | 2023-01-03 | President And Fellows Of Harvard College | Cytosine to guanine base editor |
| WO2018170184A1 (fr) | 2017-03-14 | 2018-09-20 | Editas Medicine, Inc. | Systèmes et méthodes pour le traitement d'hémoglobinopathies |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
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| EP3676376B1 (fr) | 2017-08-30 | 2025-01-15 | President and Fellows of Harvard College | Éditeurs de bases à haut rendement comprenant une gam |
| KR20250107288A (ko) | 2017-10-16 | 2025-07-11 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
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| CN110699381A (zh) * | 2019-09-17 | 2020-01-17 | 合肥瑞灵生物科技有限公司 | 地中海贫血病基因治疗载体构建方法及其用途 |
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- 2016-02-23 CN CN201680022517.8A patent/CN107532168A/zh active Pending
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| WO2014036219A2 (fr) * | 2012-08-29 | 2014-03-06 | Sangamo Biosciences, Inc. | Procédés et compositions de traitement d'un état génétique |
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| US20210009998A1 (en) | 2021-01-14 |
| CN107532182A (zh) | 2018-01-02 |
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| EP3262171A2 (fr) | 2018-01-03 |
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| SG11201706767RA (en) | 2017-09-28 |
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| AU2016225178A1 (en) | 2017-08-31 |
| AU2016225179A1 (en) | 2017-08-31 |
| WO2016135558A3 (fr) | 2016-10-20 |
| WO2016135557A3 (fr) | 2016-10-20 |
| BR112017017810A2 (en) | 2018-04-10 |
| US20210317450A9 (en) | 2021-10-14 |
| US12134767B2 (en) | 2024-11-05 |
| CN107532168A (zh) | 2018-01-02 |
| US10738305B2 (en) | 2020-08-11 |
| SG11201706766WA (en) | 2017-09-28 |
| WO2016135557A2 (fr) | 2016-09-01 |
| BR112017017812A2 (en) | 2018-04-10 |
| EP3262172A2 (fr) | 2018-01-03 |
| US20180021413A1 (en) | 2018-01-25 |
| CA2977455A1 (fr) | 2019-09-01 |
| WO2016135558A2 (fr) | 2016-09-01 |
| US20180030438A1 (en) | 2018-02-01 |
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